Arrowhead Presents Interim Data from ARO-ANG3 Phase 2 GATEWAY Study in Patients with HoFH
- 44-48% Mean Reductions in LDL-C Achieved on Top of Continued Standard of Care
- Phase 3 Planning is Ongoing
The data were presented at the 91st
Key findings from the
At study week 20, administration of 200 mg or 300 mg ARO-ANG3 on day 1 and day 84 led to the following changes:
Mean reductions in LDL-C (Martin-Hopkins) of 48.1% and 44.0%, respectively
- These reductions were achieved on top of continued standard of care, including statins, ezetimibe, PCSK9 inhibitors, and apheresis
- Mean reductions in ApoB of 39.2% and 34.5%, respectively
- Mean reductions in ANGPTL3 of 82.7% and 80.1%, respectively
- Mean reductions in LDL-C (Martin-Hopkins) of 48.1% and 44.0%, respectively
- ANPTL3 inhibition with ARO-ANG3 also reduced HDL-C, non-HDL-C, and triglycerides, consistent with published human genetic data
Safety and tolerability
- Overall, no newly identified patterns of adverse events in HoFH patients
- No treatment emergent adverse events leading to drug discontinuation, dose interruptions, or study withdrawal
- One serious adverse event of second degree atrioventricular block reported in a patient with extensive atherosclerotic cardiovascular disease history, considered not related to ARO-ANG3
About Homozygous Familial Hypercholesterolemia
Homozygous familial hypercholesterolemia (HoFH) is the most serious and rare form of familial hypercholesterolemia, which if left untreated leads to early clinical manifestations of coronary artery disease. Most cases of HoFH are due to mutations in the low-density lipoprotein receptor (LDLR) gene coding for the LDL receptor. Thus, patients with HoFH due to dysfunctional or absent LDLR can be resistant to standards of care such as statins and even resistant to alternatives such as PCSK9 inhibitors. Patients with HoFH are therefore a population with a particularly high need for additional therapy with a mechanism working outside of the LDL receptor, such as therapeutic ANGPTL3 inhibition.
About the GATEWAY Phase 2 Study
ARO-ANG3 is an investigational RNAi therapeutic designed to reduce expression of angiopoietin-like protein 3 (ANGPTL3), a hepatocyte expressed regulator of lipid and lipoprotein metabolism with multiple potential modes of action, including inhibition of lipoprotein lipase and endothelial lipase. Given the inhibitory role of ANGPTL3 in the metabolism of various lipoproteins and triglycerides, reduced expression and reduced circulating levels of ANGPTL3 may increase clearance of LDL-cholesterol, HDL-cholesterol, and triglycerides.
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